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news.Fibrocell Science (FCSC), an autologous cell and gene therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs, announced that the US Food and Drug Administration (FDA) has granted rare pediatric disease designation for Fibrocell's lead orphan gene-therapy drug candidate, FCX-007, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
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The rare pediatric disease designation augments the Orphan Drug designation granted by the FDA and announced by Fibrocell in June 2014 for FCX-007 to treat dystrophic epidermolysis bullosa (DEB), which includes RDEB.
"We are pleased that the FDA has granted our request to designate FCX-007 for the treatment of RDEB as a drug for a rare pediatric disease," said David Pernock, Chairman and Chief Executive Officer of Fibrocell.
"FCX-007 may offer RDEB patients and their families the first therapy to treat the underlying cause of the disease, bringing hope and relief to what is today a painful, disabling and often fatal congenital disorder.
"We are also pleased that the incentives offered by both the Orphan Drug and rare pediatric disease designations — including the potential to obtain a valuable Rare Pediatric Disease Priority Review Voucher from the FDA — could provide additional ways to create value for our shareholders."
The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years.
Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, upon the approval of a qualifying new drug application (NDA) or biologics license application (BLA) for the treatment of a rare pediatric disease, the sponsor of such application would be eligible for a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent NDA or BLA. The Priority Review Voucher may be sold or transferred an unlimited number of times.
FCX-007 is Fibrocell’s novel gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a gene-modified autologous fibroblast that encodes COL7 and is being developed in collaboration with Intrexon Corporation (XON), a leader in synthetic biology.
By genetically modifying autologous fibroblasts to produce COL7, ex-vivo, culturing them and then treating blisters and wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas, avoiding systemic treatment. The drug is currently in late stage pre-clinical development with an IND filing targeted for mid-2015.